Scholar Rock receives U.S. FDA expedited designation for apitegromab for treatment of patients with spinal muscular atrophy

CAMBRIDGE, Mass .– (BUSINESS WIRE) –Scholar rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on treating serious conditions in which protein growth factors play a fundamental role, today announced that the U.S. Food and Drug Administration (FDA) has granted the Fast Track designation for apitegromab, a selective inhibitor of myostatin activation, for the treatment of patients with spinal muscular atrophy (SMA). The purpose of the Fast Track designation is to facilitate the development and expedite review of drugs to treat serious conditions and to deliver new drugs to patients earlier. Through Fast Track, Scholar Rock is eligible to submit a Biologic License Application (BLA) for apitegromab if the relevant criteria are met.

“We are delighted to receive the Fast Track designation and look forward to working closely with the FDA to achieve our goal of establishing apitegromab as the first muscle-directed therapy for patients with ADS,” said Tony Kingsley, President and CEO of Scholar Rock. “We believe that the recently announced breakthrough data from the TOPAZ Phase 2 trial has shown the transformational potential of apitegromab for patients with SMA.”

Fast Track aims to address an unmet medical need defined as providing treatment where none exists or improving available therapies and is based on the fact that a drug will impact factors such as survival, daily functioning or, if left untreated, progression. to a more serious condition. In addition to the Fast Track designation, apitegromab had previously received orphan drug and rare pediatric disease designations from the FDA and the PRIME and orphan drug product designations from the EMA for the treatment of SMA.

“The recent Fast Track and PRIME designations granted by regulatory agencies highlight the unmet medical needs of patients with SMA,” said Yung Chyung, MD, medical director of Scholar Rock. “The majority of non-ambulatory patients in our TOPAZ trial experienced increases in Hammersmith scores, underscoring the therapeutic potential of apitegromab to treat motor function disorders in this patient population.”

About Apitegromab

Apitegromab is a selective inhibitor of myostatin activation and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ growth factor superfamily, is primarily expressed by skeletal muscle cells, and the absence of its gene is associated with increased muscle mass and strength in several animal species, including humans. Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote clinically meaningful improvement in motor function in patients with ADS. The United States Food and Drug Administration (FDA) has granted Fast Track Designation (FTD), Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPD), and the European Medicines Agency (EMA ) granted a Priority Drug Designation (PRIME) and Orphan Medicinal Product Designation to apitegromab for the treatment of ADS. The efficacy and safety of apitegromab have not been established and apitegromab has not been approved for use by the FDA or any other regulatory body.

About SMA

Spinal muscular atrophy (SMA) is a rare and often fatal genetic disorder that typically occurs in young children. It is estimated that 30,000 to 35,000 patients have ADS in the United States and Europe. It is characterized by loss of motor neurons, atrophy of voluntary muscles in the limbs and trunk, and progressive muscle weakness. The underlying pathology of SMA is caused by insufficient production of the motor neuron survival (SMN) protein, essential for motor neuron survival, and is encoded by two genes, SMN1 and SMN2. Although there has been progress in the development of therapeutics that treat the underlying genetic defect of SMA, via NMS-dependent pathways, there is still a high unmet need for therapeutics that directly treat SMA. muscle atrophy.

About Scholar Rock

Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative drugs for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Scholar Rock is creating a pipeline of new product candidates with the potential to transform the lives of patients with a wide range of serious illnesses, including neuromuscular disorders, cancer, fibrosis and anemia. Scholar Rock’s newly elucidated understanding of the molecular mechanisms of growth factor activation has enabled him to develop a proprietary platform for the discovery and development of monoclonal antibodies that locally and selectively target these signaling proteins at the cellular level. By developing product candidates that act in the disease microenvironment, the Company intends to avoid the historical challenges associated with inhibiting growth factors for therapeutic effect. Scholar Rock believes that the focus on biologically validated growth factors may facilitate a more efficient development path. For more information, please visit or follow Scholar Rock on Twitter (@ScholarRock) and LinkedIn (

Forward-looking statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the potential of SRK-181, Scholar’s future expectations, plans and prospects. Rock, including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of clinical trials, potential of its proprietary platform, and intellectual property protection. The use of words such as “may”, “could”, “will”, “should”, “expect”, “plan”, “anticipate”, “believe”, “estimate”, “plan”, ” intend ”,“ future ”,“ potential ”or“ continue ”and other similar expressions are intended to identify such forward-looking statements. All of these forward-looking statements are based on management’s current expectations regarding future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and unfavorably from those stated or implied by these forward-looking statements. These risks and uncertainties include the possibility that the data from the TOPAZ clinical trial may be incompatible with the data observed in subsequent clinical trials, competition from third parties who develop products for similar uses, the ability of Scholar Rock to obtain, maintain and protect its intellectual property, Scholar Rock’s dependence on third parties for the development and manufacture of product candidates, including to provide clinical trials, Scholar Rock’s ability to manage expenses and obtain additional funds as necessary to support its business operations and establish and maintain strategic business alliances and new business initiatives, and the impacts of public health pandemics such as COVID-19 on business operations, including its clinical trials, as well as more risks fully discussed in the section titled “Risk Factors” in the ra Scholar Rock’s quarterly report on Form 10-Q for the quarter ended March 31, 2021 as well as discussions of potential risks, uncertainties and other significant factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Forward-looking statements represent the views of Scholar Rock only to date and should not be taken as representative of its views at a later date. All information in this press release is as of the date of the press release, and Scholar Rock assumes no obligation to update such information except as required by law.

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